Evidence map›Paper›PMID 37804000›Full record

ArticleAnnals of clinical and translational neurology2023

Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy.

Mathula Thangarajh, Michael P McDermott, Michela Guglieri, Robert C Griggs

Open access · goldAbstract read
In one paragraph

Article in Annals of clinical and translational neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.5field-weighted citation impact, top 17% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 10 citations in OpenAlex.

  1. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 3 institutions in 2 countries.

Mathula ThangarajhDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia, USA.ORCID 0000-0001-9811-0838
Michael P McDermottUniversity of Rochester Medical Center, School of Medicine and Dentistry, Rochester, New York, USA.
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals National Health Service Foundation Trust, Newcastle, UK.
Robert C GriggsUniversity of Rochester Medical Center, School of Medicine and Dentistry, Rochester, New York, USA.
University of Rochester Medical Center · USNational Health Service · GBVirginia Commonwealth University · US

Funding

Wright Regional Center for Clinical and Translational ScienceUM1TR004360 · NCATS · VIRGINIA COMMONWEALTH UNIVERSITY · PI FREDERICK Gerard MOELLER · 2023 to 2026
$16.3M
FOR-DMD:Double-blind randomized trial to optimize steroid regimen in Duchenne MDU01NS061799 · NINDS · UNIVERSITY OF ROCHESTER · PI GRIGGS, ROBERT C, GUGLIERI, MICHELA · 2010 to 2019
$13.7M
Biostatistics and Data Management for a Trial of Corticosteroid Regimens in DMDU01NS061795 · NINDS · UNIVERSITY OF ROCHESTER · PI MCDERMOTT, MICHAEL P, TAWIL, RABI · 2010 to 2019
$3.5M
CTSA K12 Program at Virginia Commonwealth UniversityK12TR004364 · NCATS · VIRGINIA COMMONWEALTH UNIVERSITY · PI SERGE PATRICK NANASINKAM, Gretchen N Neigh · 2023 to 2026
$3.0M
PAR-18-953 (ASCOT-DMD)R21TR004007 · NCATS · VIRGINIA COMMONWEALTH UNIVERSITY · PI THANGARAJH, MATHULA · 2021 to 2022
$457k
NCATS NIH HHS K12 TR004364NCATS NIH HHS R21 TR004007NCATS NIH HHS R21TR004007NCATS NIH HHS UM1 TR004360NINDS NIH HHS U01 NS061795NINDS NIH HHS U01 NS061799
6 · The paper itself

Abstract

objectiveWe explored various prognostic factors of motor outcomes in corticosteroid-naive boys with Duchenne Muscular Dystrophy (DMD).

methodsThe associations between parent-reported neurodevelopmental concerns (speech delay, speech and language difficulties (SLD), and learning difficulties), DMD mutation location, and motor outcomes (6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA) total score, 10-meter walk/run velocity, and rise from floor velocity) were studied in 196 corticosteroid-naive boys from ages 4 to less than 8 years.

resultsParticipants with SLD walked 25.8 fewer meters in 6 minutes than those without SLD (p = 0.005) but did not demonstrate statistical differences in NSAA total score, 10-meter walk/run velocity, and rise from floor velocity. Participants with distal DMD mutations with learning difficulties walked 51.8 fewer meters in 6 minutes than those without learning difficulties (p = 0.0007). Participants with distal DMD mutations were slower on 10-meter walk/run velocity, and rise from floor velocity (p = 0.02) than those with proximal DMD mutations. Participants with distal DMD mutations, who reported speech delay or learning difficulties, were slower on rise from floor velocity (p = 0.04, p = 0.01) than those with proximal DMD mutations. The mean NSAA total score was lower in participants with learning difficulties than in those without (p = 0.004).

interpretationCorticosteroid-naive boys with DMD with distal DMD mutations may perform worse on some timed function tests, and that those with learning difficulties may perform worse on the NSAA. Pending confirmatory studies, our data underscore the importance of considering co-existing neurodevelopmental symptoms on motor outcome measures.

Indexed as

Language Development DisordersMuscular Dystrophy, DuchenneAdrenal Cortex HormonesHumansMaleOutcome Assessment, Health CareWalkingAdrenal Cortex Hormones

Identifiers

PMID37804000
PMCPMC10723228
OpenAlexW4387420623

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.